Not recruitingRare disease

Study on Ponatinib for Achieving Treatment-Free Remission in Patients with Chronic Myeloid Leukemia

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a type of blood cancer called chronic myeloid leukemia (CML), specifically in its chronic phase. The treatment being tested is a medication called Ponatinib, which is taken as a film-coated tablet. Ponatinib is a type of drug known as a tyrosine kinase inhibitor, which works by blocking certain proteins that help cancer cells grow.

The purpose of the study is to assess patients who have achieved a specific level of response, known as MR4, after two years of treatment with Ponatinib. MR4 is a term used to describe a deep molecular response, indicating a very low level of cancer cells in the body. The study will follow participants over a period of 24 months to monitor their response to the treatment. During this time, researchers will evaluate the molecular status of the disease, the safety and tolerability of Ponatinib, and any side effects that may occur. Additionally, the study will look at the quality of life of participants while on the treatment.

Throughout the study, researchers will also identify factors that may be associated with achieving MR4 or better, such as risk scores and the duration of previous treatments. The study aims to provide insights into the potential for treatment-free remission, where patients may be able to stop treatment while maintaining their response. Overall survival and progression-free survival will also be evaluated to understand the long-term benefits of Ponatinib therapy.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initiation of treatment
Upon joining the study, the patient begins treatment with ponatinib, which is administered in the form of Iclusig 15 mg film-coated tablets.
The medication is taken orally, following the prescribed dosage and schedule provided by the healthcare team.
Step 2
Treatment phase
The patient continues to take ponatinib for a duration of 24 months.
During this period, the patient's response to the treatment is monitored, focusing on achieving a molecular response known as MR4.
Step 3
Evaluation of molecular status
Throughout the treatment, the patient's molecular status is evaluated at scheduled visits.
The evaluations include checking for different levels of response, such as no MMR (major molecular response), MMR, MR4, and MR4.5.
Step 4
Monitoring safety and quality of life
The safety profile and tolerability of ponatinib are assessed regularly to identify any adverse events.
The patient's health-related quality of life is also monitored to ensure well-being during the treatment.
Step 5
Identification of factors for response
Clinical and biological factors associated with achieving MR4 or better are identified.
Factors such as risk scores, gender, duration of previous treatments, and molecular level at study entry are considered.
Step 6
Evaluation of survival outcomes
The study evaluates overall survival and progression-free survival as part of the treatment outcomes.
The potential for a treatment-free remission (TFR) approach is also assessed.

Who can join the trial?

6 criteria

Must be a female or male who is **18 years of age or older**.
Must have **chronic myeloid leukemia (CML)** in the **chronic phase**. This is a type of blood cancer that is in a specific stage.
Must have a **BCR-ABL IS** level between **0.5-0.01**. This is a specific measurement from a blood test called **PCR** that checks for certain genetic markers.
Must not have achieved **MR4** or a stable **MR4** after at least **3 years** of treatment with certain medications like **nilotinib**, **dasatinib**, or **bosutinib**. **MR4** is a specific level of response to treatment, indicating a very low level of cancer cells.
Must have **Philadelphia chromosome** and/or **BCR-ABL fusion gene** positive CML. These are specific genetic changes found in the cancer cells.
Must have had an **eye examination**, including a check of the back of the eye (**fundoscopy**), by an eye doctor within **8 weeks** before starting the treatment.

Who cannot join the trial?

4 criteria

Patients who have not achieved MR4 after 2 years of treatment with ponatinib. MR4 means a specific level of response to treatment where the disease is very well controlled.
Patients who are not in the chronic phase of chronic myeloid leukemia. The chronic phase is an early stage of the disease where symptoms are usually mild.
Patients who are not within the specified age range for the study.
Patients who belong to a group that is considered vulnerable, meaning they might need special protection or care.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Klinikum Bayreuth GmbH
Bayreuth, Germany
Verified
Universitaetsklinikum Aachen AöR
Aachen, Germany
Verified
Universitaetsklinikum Heidelberg AöR
Heidelberg, Germany

Trial locations

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Investigated drugs

Ponatinib is a medication used in this clinical trial to treat patients with chronic myeloid leukemia. The trial aims to assess patients who have achieved a specific level of remission, known as MR4, after two years of treatment with this medication. Ponatinib works by blocking certain proteins that promote the growth of cancer cells, helping to control the disease.

Ponatinib

What is already known about the treatment

Ponatinib – Ponatinib is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in achieving treatment-free remission in patients with chronic myeloid leukemia in the chronic phase. The main therapeutic indication for Ponatinib is chronic myeloid leukemia, particularly for patients who have achieved a significant molecular response after two years of treatment. At the molecular level, Ponatinib works by inhibiting specific proteins that promote the growth of cancer cells, particularly targeting the BCR-ABL protein. It is classified as a tyrosine kinase inhibitor, a type of medication that blocks the action of enzymes involved in the signaling pathways that lead to cancer cell growth.

Investigated diseases

Chronic Myeloid Leukemia in Chronic Phase – This is a type of cancer that affects the blood and bone marrow. It is characterized by the overproduction of white blood cells. In the chronic phase, the disease progresses slowly and may not cause noticeable symptoms initially. Over time, the number of abnormal cells increases, which can lead to fatigue, weight loss, and an enlarged spleen. The chronic phase can last for several years before potentially advancing to more aggressive phases. Monitoring and regular check-ups are essential to track the progression of the disease.